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Open Access Research

Impact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany

Paola Giunti1*, Julia Greenfield2, Alison J Stevenson2, Michael H Parkinson1, Jodie L Hartmann3, Ruediger Sandtmann4, James Piercy5, Jamie O’Hara5, Leo Ruiz Casas5 and Fiona M Smith5

Author Affiliations

1 Department of Molecular Neuroscience, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK

2 Ataxia UK, 1-3 Brixton Road, London, UK

3 Takeda Pharmaceuticals Europe Ltd, 61 Aldwych, London, UK

4 Takeda Pharma GmbH, Aachen, Germany

5 Adelphi Real World, Bollington, Cheshire, UK

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Orphanet Journal of Rare Diseases 2013, 8:38  doi:10.1186/1750-1172-8-38

Published: 28 February 2013

Abstract

Background

Friedreich’s Ataxia (FRDA) is a neurodegenerative disorder that causes progressive damage to the central and peripheral nervous systems having a significant impact upon quality of life. With little information in the literature, cross-sectional observational studies were conducted in the UK and Germany to collect data on resource use and the burden of the disease on individuals and their caregivers.

Methods

Cross-sectional observational studies were conducted in the UK and Germany to estimate the burden of FRDA on individuals and on the respective healthcare systems. A total of 75 individuals in the UK and 28 in Germany were recruited to the study. Participants in both countries were asked to complete a Patient and Caregiver Information Form (PCIF), regarding access to, and use of, healthcare resources, and the impact FRDA has on their lifestyle. In Germany, doctors were asked to complete a Patient Record Form (PRF). Analyses of annual direct and indirect resource utilization were conducted for both countries while costs were calculated for the UK only. These figures were compared to the costs associated with Parkinson’s disease; one of the most common neurodegenerative conditions and the one most similar in terms of disease progression.

Results

The results showed that the annual burden of FRDA is significant and falls on the health and social care sectors, on society, on caregivers and on the individuals themselves. In the UK FRDA had a total annual cost per person of between £11,818 and £18,774 depending on whether the cost of long-term unemployment was included.

Typically the largest component of direct costs is associated with professional care. Given the high proportion of children and young adults recruited and the long disease duration, (typically 40-50 years for FRDA, compared with 20 years for Parkinson’s disease), these figures may underestimate the true burden of the disease.

Conclusion

It is hoped that these estimates of resource utilization, can help in understanding the previously unquantified burden of FRDA. Given the long disease duration, management strategies should seek to minimise the impact of the condition on individuals and their caregivers, while maximising quality of life.

Keywords:
Ataxia; Friedreich’s Ataxia; Quality of life; Cost of illness; Resource utilization